August 13, 2002
To: BCCNS Life Support Network Members
We are writing again about our clinical trial that is testing the possible efficacy of Celebrex in reducing the number of basal cell carcinomas that develop in patients with the basal cell nevus syndrome. Since opening the Study in May of 2001 we have enrolled approximately half of the 60 patients that we need. This number was chosen to give us a very good chance of seeing any real effects that the drug has ~ if we study fewer patients, then we reduce drastically the chances of actually knowing for sure whether or not it works. We wrote to you in March with two thoughts about the Study, and both still are foremost in our minds.
The first is that we remain humbled and inspired by the dedication that trial participants have shown. Some have driven many hours each way to visit us in San Francisco or in New York; some have come by air and stayed overnight. We have tried hard to gather more funds so that we can raise the reimbursement for expenses, but we do not yet know whether these efforts will be successful.
The second is that we remain VERY anxious to recruit the 25 more patients that we need to complete the enrollment in this trial. We still are absolutely convinced of the importance of the Study. We think that it is important because we hope that this drug actually might reduce the number of new tumors that appear. But beyond that hope for this trial, we hope that this will be but the first of a series of trials that eventually will make a real difference in the lives of patients with NBCCS. Again, the National Cancer Institute made a very serious commitment of funds to us for a five year period, and if we are not successful in recruiting as many patients as we had predicted, then for sure they will not be willing to fund future efforts to test new agents.
So if you think there is even a remote possibility that you might be able to participate in our trial, now is the time to call us to discuss what is needed and whether you might be eligible. Together we have a chance of improving the lot of BCCNS patients everywhere. We always would be happy to have you call us with any suggestions, comments – or anything else that you might like to discuss. It’s 1-800-386-1001
Best wishes,
Ervin H. Epstein, Jr. MD
University of California / San Francisco
David Bickers, MD
Columbia University
Dear BCCNS Life Support Network Members:
We are writing now to give you an update regarding our clinical trial that is testing the possible efficacy of Celebrex in reducing the number of basal cell carcinomas that develop in patients with the basal cell nevus syndrome. As you know, we have been working on this clinical Study for the past 3 years and began enrolling patients in May, 2001. So far, we have enrolled 22 patients, and it is much too early to tell whether or not the Celebrex has had any effect, especially since we who are seeing the patients do not ourselves know who is getting the Celebrex and who is getting the placebo ("sugar pill"). Furthermore, even our "unblinded" colleagues can not tell whether or not Celebrex is having an effect because we have not enrolled anywhere near the number that we need to be able to get scientifically certain data - that number is 60 persons.
Two thoughts now as we are part way into this Study.
We have been humbled and inspired by the dedication that some people have shown in participating in this trial. Some have driven 8 or even 14 hours each way to visit us in San Francisco or in New York; some have come by air and stayed overnight, often spending their own money since our available reimbursements do not match the expenses that patients from more distant cities are incurring.
At the same time we are disappointed that we have had so much trouble recruiting patients to participate. We are disappointed because we are very excited about the possibility of improving the care of BCNS patients but obviously we have not been able to communicate that excitement to the larger number of BCNS patients who have chosen not to enroll in this Study. We want to stress that we do understand that the constraints of the Study - having to promise to come to New York, Cleveland, San Francisco, or Los Angeles every three months for three years, avoiding various other drugs, getting blood tests, etc. - are daunting to some and even make it impossible for others. So for some, no matter how much they might like to participate, it will be impossible for them to do so.
But we also want to stress how important we think this Study is. The National Cancer Institute made a very serious commitment of funds to us for a five year period. These funds are partially for laboratory-based studies, and indeed we have some thousands of mice and a dozen lab persons working to understand BCNS better and to develop newer therapies and preventive measures based on the results in the lab. But the centerpiece of the project is the clinical trial. We are now in the third of the five years of this funding, and we must think seriously about how we can convince the National Cancer Institute to agree to provide another five years of funding. We are quite certain that the very best way to assure continued funding is to show them that we can enroll enough patients to get meaningful results. We do not know whether or not Celebrex will be able to prevent the development of BCCs - that is why we are doing this costly and complicated study. We are committed to making this a long-term project - to testing other agents after we are done with this one. But without evidence of our ability actually to conduct such studies, we fear that it will be impossible to gain funding to continue this important research.
We want to emphasize that patients who take the placebo ("sugar pill") make every bit as great a contribution to our all finding out whether or not the drug helps as do those who take the Celebrex. We can not promise that the drug will help but we can promise careful skin examinations every three months and the possibility that you will help not only others with this disease in the future but, who knows, maybe you and other members of your family with BCNS.
So, yes we urge you to think seriously about enrolling in this Study. Individually no one is going to find great breakthroughs that change the burden of this disease. Together there is at least a chance that we all can participate in such an advance.
Thanks for your support. Please do not hesitate to call us (800-386-1001) if you have any questions about this Study or about other aspects of our research program.
Best wishes.
Sincerely yours,
Ervin Epstein, Jr., M.D., and David Bickers, M.D.